In Wolfram syndrome, insulin-producing beta cells in the pancreas and brain cells selectively degenerate. Why? We extensively studied their skin cells and lymphocytes, and did not find any problems although these cells have impaired WFS1 functions. I always think that this mysterious finding will eventually lead to a cure for Wolfram. If we understand why beta cells and brain cells are sensitive to impaired Wolfram gene functions, we can design a new treatment.
We got encouraging results last week. It seems like that the same drug can potentially restore functions in beta cells and brain cells of Wolfram patients. Our results suggest that pancreatic beta cells and brain cells degenerate by the same mechanisms. Thus, a drug that can stop the degeneration of beta cells should be able to stop the degeneration of brain cells and eye cells. I was really encouraged. We should keep on moving forward.
I hope you will have a wonderful Sunday. I am sending kind thoughts toward you.